Today we learned that the FDA will host an Advisory Committee (Ad Comm) meeting on November 24 to discuss the NDA for BioMarin’s drisapersen compound. Tomorrow the official notification is expected in the Federal Register. Once the official notification is published, PPMD will share important information with you regarding how you can participate.
We reached out to officials at the FDA to express our community’s eagerness to learn the details for the Sarepta Ad Comm which was expected to be announced for around the same time period. Moments later, we spoke to Ed Kaye from Sarepta and the company put out a press release that announced they are tentatively schedule to participate in an Advisory Committee meeting on January 22, 2016. The November 23 date that we assumed would be dedicated to an eteplirsen Advisory Committee meeting was no longer on the FDA calendar.
Why was the date changed? There is a reasonable answer based on the timing of the submissions of both companies. Biomarin submitted their package to FDA in May 2015 and Sarepta in July 2015. Once a submission occurs, a clock is started. FDA must notify the respective company within 60 days about their intent to ‘file’. In other words, FDA notifies the company that they intend to review the package submitted. Once the notification of the intent to file is given, FDA has 6 months to complete the review and render a decision. We assume that from the FDA viewpoint, the decision to separate the Advisory Committee meetings is within the legal framework of the review process.
We all have many questions about what the January AdComm date for Sarepta, after the PDUFA date for BioMarin may mean. And PPMD is working with our regulatory experts to seek those answers and to understand how we can continue to work together as a community to ensure that both products are offered regulatory pathways to success so that patients ultimately may have access to either product, if approved.
PPMD believes all patients need and deserve options. Patients and their families seek out clinicians to discuss options. The decisions we face now have the potential to redefine a person, preserve function, and potentially save a life. We believe that our clinicians and our families need as much data as possible to make these decisions and for this reason, were thrilled with the possibility of back-to-back Advisory Committee meetings. We wanted and needed to learn the data, hear questions from the AdComm, listen to the discussion, and participate by telling our side of the story, our experience with these compounds, and our urgent need for therapies.
Together, the community will reach out to FDA. Whether or not we will be able to encourage a change in scheduling of Advisory Committee meetings is difficult to predict. But we do want to voice our concern to FDA and restate the urgent need for approvals.
PPMD will continue to work closely with both BioMarin and Sarepta, doing everything we can to help them prepare for their anticipated Advisory Committee meetings in the coming weeks.
We believe both drugs deserve an approval. Patients with Duchenne deserve therapies.
And PPMD is working closely with the community, with you. We are listening to your questions and are doing everything we can to get the answers we all deserve. As always, we are here for you and we will navigate this unchartered territory together.